Figure 1.

Splicing of EGFP-IVSI-110 mRNA. Schematic diagram illustrating EGFP pre-mRNA containing intron I of ^huβ-globin carrying the G>A splicing mutation at nucleotide 110. The mutation activates an aberrant splice site. Short bars at position 86 and 110 of intron I represent targeted 2’-O-Me phosphorothioate antisense oligonucleotide (AOs), which have been targeted to redirect aberrant splicing. Primers Fwd E 1-110 and Rev E 1-110 were used in RT-PCR